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โ† Trials
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Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy

RecruitingEARLY Phase 1Ages 4 Years - 8 YearsMaleShanghai Jiao Tong University School of Medicine
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In plain words

What the trial is, and whether it could suit you

The purpose of the study is to assess the safety, tolerability, and efficacy of BBM-D101 to treat patients with Duchenne Muscular Dystrophy.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria: 1. The legal guardian of the subject fully understands the purpose, nature, methods, and possible risks of the study, and signs a written informed consent form; 2. The study includes ambulatory male subjects who are at least 4 years old and less than 8 years old (4 years old โ‰ค age \< 8 years old) ; 3. Genetically confirmed diagnosis of DMD; 4. Have at least 1 of the following typical clinical signs or laboratory abnormalities of DMD: proximal muscle weakness, waddling gait, pseudo gastrocnemius hypertrophy, Gower\'s sign, pterygoid scapula; 5. Ability to cooperate with motor assessment testing, magnetic resonance imaging (MRI) and muscle biopsy according to the requirements of the study. Exclusion Criteria: 1. Hepatitis B surface antigen (HBsAg) positive, hepatitis B virus deoxyribonucleic acid (HBV-DNA) โ‰ฅ1000U/mL, hepatitis C virus ribonucleic acid (HCV-RNA) positive or human immunodeficiency virus (HIV) positive; 2. Receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc.; 3. Left ventricular ejection fraction (LVEF) \<50% or โ‰ฅ class III cardiac function defined by New York Heart Association (NYHA); 4. With severe or persistent arrhythmias and congenital heart disease. 5. The subject\'s preventive treatment/cardiomyopathy treatment changes within 1 month before the start of the study treatment; 6. With underlying liver disease, such as previous diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy, or hepatic fibrosis โ‰ฅ stage 3; or nodules, cysts found by B-ultrasound in the past, or elevated alpha-fetoprotein in laboratory tests during the screening period, etc., and these abnormalities are judged by the investigator to be clinically significant;
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What it tests

  • BBM-D101
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Where

1 site
Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, China
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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